The Why Files -- whyfiles.org

Stem cells — legal confusion!

The Michael J. Fox Foundation for Parkinson's Disease has contributed $50 million towards research on this brain disease. Fox is shown during a visit to the UW-Madison campus, where his foundation has contributed $1.2 million for human ES research.

The old stem cell battles came roaring back on Aug. 23, when a federal judge blocked an executive order that expanded federal funding for embryonic stem cell research. District Judge Royce Lamberth held the 2009 order from President Obama violated a long-standing ban on federal funding for research that destroyed human embryos.

That hold has, in turn, since been stayed by an appeals court, reflecting a fast-changing legal situation that has embryonic stem cell researchers as nervous as a kitten at a dog pound.

In the Washington, D.C. District Court, two pro-life adult-stem cell researchers convinced Judge Lamberth that the Dickey-Wicker budget amendment explicitly prohibits embryonic stem cell (ES) research because it, and we quote, bans federal funding for "research in which a human embryo or embryos are destroyed, discarded, or knowingly subjected to risk of injury or death greater than that allowed for research on fetuses in utero."

Lamberth's preliminary injunction seems even more restrictive than the 2001 policy of President George W. Bush, which allowed funding for research on several specific types of human ES cells. Under the Bush policy, researchers had to build separate, privately funded labs with duplicate equipment for ES research on other ES cells.

Obama's order loosened that restriction, while retaining safeguards, such as prohibiting the purchase of embryos, and was considered to end the political debate -- until last month.

An argument based on hope - or hype?

Advocates of human ES cell research say the amendment, written in 1996 before human ES cells had even been identified, was aimed at preventing human cloning and should not be used to stymie life-saving research into versatile stem cells. They add that the human embryos that provided the ES cells under study were surplus at fertility clinics and would have been destroyed long ago.

To opponents, destroying embryos amounts to destroying human life.

Slowly, despite eight years of tight federal restrictions, the science is moving forward. This August, the first human test of cells grown from ES cells was approved. The trial will test whether it's safe to inject cells that support and stimulate nerve cells in the spinal cord into people paralyzed by a recent injury. If the injections prove safe, the researchers will test whether these ES-derived cells can help damaged spinal cords go back to work and restore movement.

Still promising after all these years

Keep 'em guessing!

On Sept. 9, an appeals court stayed Lamberth's decision, and ordered both sides to submit briefs on reinstating the injunction by Sept. 20. Although the National Institutes of Health is again reviewing ES cell grants and disbursing funds, "I take no solace in the ruling because so much uncertainty remains about the future of human stem cell research," George Daley, a stem cell researcher at Children's Hospital Boston, told the Washington Post.
The plaintiff's lawyer asked for summary judgment to "make it clear to NIH that not only can it not provide further grants for additional embryonic stem cell research with taxpayer dollars, but that scientists who already received, but have not spent, tax dollars should not spend the remainder of the funds until the case is concluded."

Human embryonic stem cells mature into every cell type, and "could be useful for studying or treating a wide variety of conditions, from developmental disabilities like Down syndrome to degenerative diseases like Parkinson's, ALS, Huntington's disease, heart disease, even juvenile diabetes, blindness and hearing loss," says cardiologist Timothy Kamp, director of the Stem Cell and Regenerative Medicine Center at the University of Wisconsin-Madison.

Human ES cells are used to test drugs and explore the fundamental question of how primitive cells morph into their specialized adult forms.

Lamberth's ruling caused a wave of confusion among researchers who hope ES cells can lead to treatments or cures. "There's turmoil, consternation, frustration," says Lawrence Goldstein, a professor of cellular and molecular medicine at the University of California San Diego. "We are trying to run labs, to get research done, and this is a very confusing time, it puts a lot of important research funding in jeopardy. This is not about the scientists, it's about the people who have diseases that we are trying to develop therapies for."

"This is detrimental on so many levels," says David Gamm, an assistant professor of ophthalmology at UW-Madison who studies irreversible forms of blindness. "People don't realize it even affects the 'Bush' lines [cells that could be studied with federal money under Bush]; it says the federal government will not provide money for anything to do with ES cells." Even people who study adult stem cells are hobbled, Gamm says, because they often must be compared to ES cells. "This is crippling on many levels."

From the patient's side

The federal decision nettled organizations and individuals concerned with a range of serious diseases. The American Diabetes Association, for example, says, "Scientists from across the United States and throughout the world ... believe that stem cell research, especially embryonic stem cell research, holds great promise in the search for a cure and better treatments for diabetes."

Courtesy Stephen Byer

Ben Byer (1971-2008), shown with his son John in 2007, died of ALS -- a disease that may someday be treated with embryonic stem cells.

Psychiatrist Stephen Levick, of the University of Pennsylvania, who closely follows diabetes, prefers biology to technology. "You could argue that so much progress is being made in type I diabetes, with the insulin pump, continuous glucose monitoring, and eventually an artificial pancreas, but this is like improving the iron lung [used to keep polio victims alive before polio vaccine was invented]. The ideal treatment for type 1 diabetes, and for many other illnesses, is biological, where the damaged or diseased cells or tissues are healed or replaced by cells that work normally. Millions of years of evolution has perfected those cells. Why not go for that solution?"

The National Multiple Sclerosis Society "supports all promising avenues of research that could lead to stopping the progression, repairing the damage or ending MS -- including expanding the number of stem cell lines that are available for federally funded research. Alleviating restrictions on the use of embryonic stem cells represents an important step that will allow us to move forward..."

Families affected by always-fatal nerve disease ALS (Lou Gehrig's disease) do not have time to fight the funding shutdown, says Stephen Byer, a patient advocate and educator. After a recent visit to five ALS families in the New York area, he told us, "Without exception, they said the same thing: 'This means we will have to continue going to other countries to get [adult] stem cell therapy. What did you learn during your visit to BrainStorm or Cellular Technology in Israel? What will it cost? How are we going to get dad to Tel Aviv with a respirator and a wheelchair?'"

Granted, even if ES funding is restored, real treatments for ALS remain years away, but they may offer a more complete solution, Byer says. "Adult stem cell therapy works for a while, but you have to keep repeating it. But it's better than watching someone die."

One source of hope for ALS patients comes from the Goldstein lab in San Diego, which is growing human embryonic stem cells into support cells in the brain and spinal cord. The money comes from the state-funded California Institute of Regenerative Medicine, Goldstein says, and is not subject to the federal cutoff, "but one would like to see federal funding of comparable projects. We don't have a corner on the market for bright ideas."

Under a fume hood, a technician feeds stem cells in the lab of embryonic stem cell discoverer James Thomson.

Embryonic vs. adult?

The scientists who sued to stop federal funding claimed to have "legal standing" on the grounds that funding for their specialty, adult stem cell research, is crimped when ES research is also eligible for money. Yet in 2011, the National Institutes of Health plans to spend $155 million on human adult stem cell research, and just $126 million for human ES cells.

Although adult stem cells can be grown into a wide variety of cells, they are not a perfect replacement for ES cells, says Stephen Duncan, director of regenerative medicine and stem cell biology at the Medical College of Wisconsin. "There is a feeling that we don't need to work with embryonic stem cells, because adult stem cells can do everything that they can do. But adult stem cells, after 50 years, can only be used to treat blood disorders," such as for bone-marrow transplant after cancer treatment.